Rett syndrome
Recent articles
New RNA editor boasts increased versatility, safety
The “PRECISE” technique reprograms cells in a way that, unlike DNA editors, avoids potentially permanent off-target effects.
![A digital rendering of a strand of RNA](https://www.thetransmitter.org/wp-content/uploads/2024/04/1200-programmable-RNA-trans-splicing-transmitter-neuroscience-1024x683.webp)
New RNA editor boasts increased versatility, safety
The “PRECISE” technique reprograms cells in a way that, unlike DNA editors, avoids potentially permanent off-target effects.
New template streamlines sharing clinical trial results
This month’s Going on Trial newsletter explores a new tool for communicating with clinical trial participants and their families, among other drug development news.
![Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.](https://www.thetransmitter.org/wp-content/uploads/2023/01/844-going-on-trial-autism-drugs-02.jpg)
New template streamlines sharing clinical trial results
This month’s Going on Trial newsletter explores a new tool for communicating with clinical trial participants and their families, among other drug development news.
Trials test utility of EEG biomarkers for autism-related conditions
This month’s Going on Trial newsletter dives into an electroencephalography biomarker that could track the efficacy of treatments for dup15q and Angelman syndromes, among other drug development news.
![Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.](https://www.thetransmitter.org/wp-content/uploads/2023/01/844-going-on-trial-autism-drugs-02.jpg)
Trials test utility of EEG biomarkers for autism-related conditions
This month’s Going on Trial newsletter dives into an electroencephalography biomarker that could track the efficacy of treatments for dup15q and Angelman syndromes, among other drug development news.
How organ-on-a-chip models can help drug development
This month’s Going on Trial newsletter explores how organ-on-a-chip models could smooth the transition from preclinical to clinical trials, among other drug development news.
![A hand reaches from above to add a pill to a stack that is resting against the x-axis of a graph.](https://www.thetransmitter.org/wp-content/uploads/2023/01/844-going-on-trial-autism-drugs-01.jpg)
How organ-on-a-chip models can help drug development
This month’s Going on Trial newsletter explores how organ-on-a-chip models could smooth the transition from preclinical to clinical trials, among other drug development news.
Going on Trial: Gene therapy for Rett; return to arbaclofen
This month’s newsletter looks at the early safety data from the first gene therapy trial for Rett syndrome, among other drug development news.
![Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.](https://www.thetransmitter.org/wp-content/uploads/2023/01/844-going-on-trial-autism-drugs-02.jpg)
Going on Trial: Gene therapy for Rett; return to arbaclofen
This month’s newsletter looks at the early safety data from the first gene therapy trial for Rett syndrome, among other drug development news.
The future of autism therapies: A conversation with Lilia Iakoucheva and Derek Hong
If a therapy for autism’s core traits makes it to market, it will likely take one of three forms, the researchers say.
![Illustration of a room with DNA sequence wallpaper and three doors leading to abstract new environments.](https://www.thetransmitter.org/wp-content/uploads/2023/04/844-lede-future-of-autism-treatments.jpg)
The future of autism therapies: A conversation with Lilia Iakoucheva and Derek Hong
If a therapy for autism’s core traits makes it to market, it will likely take one of three forms, the researchers say.
FDA approval of trofinetide may spur further drug development for Rett
The drug, welcomed by patients, might be just the first of many.
![Drop of liquid medicine with air bubbles is dripping from a pipette on a light pink background.](https://www.thetransmitter.org/wp-content/uploads/2023/03/844-trofinetide-lede.jpg)
FDA approval of trofinetide may spur further drug development for Rett
The drug, welcomed by patients, might be just the first of many.
Debut drug for Rett syndrome at edge of approval
The U.S. Food and Drug Administration plans to make an approval decision on the first-ever drug for girls and women with Rett syndrome by 12 March.
![Illustration of a gloved hand reaching down to place a pill bottle on a pedestal.](https://www.thetransmitter.org/wp-content/uploads/2023/03/844-enterprise-rett.jpg)
Debut drug for Rett syndrome at edge of approval
The U.S. Food and Drug Administration plans to make an approval decision on the first-ever drug for girls and women with Rett syndrome by 12 March.
Going on Trial: Orphan drugs; CBD; bumetanide
This month’s newsletter takes a close look at the orphan drug program in the United States, several cannabis-based therapies and a secondary analysis of bumetanide, among other new developments in autism-related drug trials.
![Illustration of a sheet of red and white pills, with the red pills arranged in the form of a question mark.](https://www.thetransmitter.org/wp-content/uploads/2023/01/844-going-on-trial-autism-drugs-02.jpg)
Going on Trial: Orphan drugs; CBD; bumetanide
This month’s newsletter takes a close look at the orphan drug program in the United States, several cannabis-based therapies and a secondary analysis of bumetanide, among other new developments in autism-related drug trials.
Biotech downturn hurts companies targeting autism-linked conditions
After a year of intense growth, funding for biotech is in decline. The result is layoffs and program cuts — and maybe some innovation.
![Illustration of a series of pills in petri dishes laid out to look like a descending line graph.](https://www.thetransmitter.org/wp-content/uploads/2023/02/844-final.jpg)
Biotech downturn hurts companies targeting autism-linked conditions
After a year of intense growth, funding for biotech is in decline. The result is layoffs and program cuts — and maybe some innovation.
Explore more from The Transmitter
Cell population in brainstem coordinates cough, new study shows
The work also adds to a growing body of evidence showing that mice, and their genetic toolbox, can be used to study cough.
![](https://www.thetransmitter.org/wp-content/uploads/2024/09/1200_coughingmice-1024x720.webp)
Cell population in brainstem coordinates cough, new study shows
The work also adds to a growing body of evidence showing that mice, and their genetic toolbox, can be used to study cough.
In updated U.S. autism bill, Congress calls for funding boost, expanded scope
The current Autism CARES Act sunsets in late September.
![Capitol building](https://www.thetransmitter.org/wp-content/uploads/2024/09/capitolhill-1200-1024x692.webp)
In updated U.S. autism bill, Congress calls for funding boost, expanded scope
The current Autism CARES Act sunsets in late September.
Ketamine targets lateral habenula, setting off cascade of antidepressant effects
The drug’s affinity for overactive cells in the “anti-reward” region may help explain its rapid and long-lasting results.
![Illustration of ketamine blocking open ion channels in active NMDA receptors, quieting the cells and disrupting downstream signaling involved in depression.](https://www.thetransmitter.org/wp-content/uploads/2024/09/1200-ketamine-receptor-transmitter-neuroscience-1024x683.webp)
Ketamine targets lateral habenula, setting off cascade of antidepressant effects
The drug’s affinity for overactive cells in the “anti-reward” region may help explain its rapid and long-lasting results.